A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
Start Date
4/16/2024
Completion Date
1/15/2029
Summary
US, multicenter, cohort, open label observational study with primary data collection. Ancillary protocol-specified procedures to address the study objectives (eg, assessment of ADA) may be considered outside the standard of care for acid sphingomyelinase deficiency (ASMD), but the study methodology remains non-interventional, as the additional collection of data from participants will not dictate treatment. The total overall study duration will be 5 years. The follow-up period will be a minimum of 1 year to a maximum of 3 years. The enrollment period will be up to 4 years, to allow a minimum of 1 year of follow-up for the last participant enrolled.
Detailed Description
This is a hybrid study design. Patients need not travel to existing study sites in order to enroll.
Eligibility Criteria
Age Range: No minimum to 2 years
Interventions
Olipudase alfa
Conditions
Locations
Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002
Chicago, Illinois 60611
United States
Children's Hospital Medical Center- Site Number: 001003
Cincinnati, Ohio 45229-3039
United States
Nationwide Children's Hospital - PIN- Site Number : 1001-1
Columbus, Ohio 43205-2664
United States
Pulse InfoFrame US Inc.- Site Number: 001001
Philadelphia, Pennsylvania 19104
United States
Dell Children's Medical Center- Site Number : 1001-2
Austin, Texas 78723
United States